A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
Completed · NCT03597399
Conditions: Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
At a glance
- Research payments reported, 2025
- $136,268
- U.S. investigators named
- 9
- Sites paid
- 9
- Planned or actual enrollment
- 87
About the trial
- Sponsor
- Spark Therapeutics, Inc.
- Tested
- AAV2-hRPE65v2,voretigene neparvovec-rzyl (biological)
- Dates
- Started 2019-01-10; completion 2025-06-30
- Results posted
- Not yet
The objective of this study is to collect long-term safety information (i.e., for 5 years after treatment) associated with voretigene neparvovec-rzyl (vector and/or transgene), its subretinal injection procedure, the concomitant use of corticosteroids, or a combination of these procedures and products. The enrollment period will last for two years from the first treatment following product approval (through 31March2020) and include a minimum of 40 patients.
From ClinicalTrials.gov, last updated 2025-09-22. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Spark Therapeutics, Inc. reported $136,268 in research payments for this study in 2025 (18 payments).
Products named: Luxturna
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Aaron Nagiel, M.D., PH.D. Ophthalmology · Ventura, CA
- Byron L. Lam, MD Ophthalmology · Miami, FL
- Stephen R. Russell, MD Ophthalmology · Iowa City, IA
- Jason Comander, M.D., PH.D. Ophthalmology · Boston, MA
- Cagri G. Besirli, M.D. Ophthalmology · Ann Arbor, MI
- Robert A. Sisk, MD Ophthalmology · Cincinnati, OH
- Paul Yang, M.D., PH.D. Ophthalmology · Portland, OR
- Tomas S. Aleman, MD Retina Specialist · Philadelphia, PA
- Brigid K. Marshall, MD Retina Specialist · Nashville, TN
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Massachusetts Eye and Ear Infirmary -, MA $44,252
- Children's Hosp of Los Angeles, CA $24,413
- University of Michigan Health System, MI $19,795
- Ohsu Hospital and Clinics, OR $17,071
- Children's Hospital of Philadelphia, PA $10,778
- Baylor College of Medicine $9,809
- University of Miami $5,526
- Cei Physicians Psc Inc $3,038
- University of Iowa Hospital & Clinics, IA $1,588
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.